Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A

• Status: Completed
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Pharnext SA

Criteria

Inclusion Criteria:

• DNA proven CMT1A

• Muscle weakness in at least foot dorsiflexion (clinical assessment)

• Age between 18 and 65 years

• Male or non pregnant, non breastfeeding female

• CMT neuropathy score at screening ≤ 20

• Agrees to perform electrorophysiological studies and two cutaneous biopsies for determination of PMP22 expression and histology

• Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits

Exclusion Criteria:

• Patients with another neurological disease

• Patients using unauthorized concomitant treatments, ascorbic acid, opioids, levothyroxine and potentially neurotoxic drugs. Patients who can/agree to stop these medications 4 weeks before randomization can be included

• Patients who have participated in another trial of investigational drug within the past 30 days

• Concomitant major systemic disease

• Clinically significant history of unstable medical illness over the last 30 days (unstable angina…)

• History of significant hematologic, kidney, liver disease, or insulin-dependent diabetes

• Clinically significant abnormalities on the prestudy laboratory evaluation, physical evaluation, electrocardiogram (ECG)

• ASAT/ALAT levels above the upper limit of normal (ULN). However, patients with an isolated elevation of either ASAT or ALAT (<1.5 ULN) can be included at investigators" discretion if the remaining liver function tests are normal and if ASAT or ALAT value is stable at 2 distinct evaluations in the month prior to inclusion

• Serum creatinine levels above the upper limit of normal

• Limited mental capacity or psychiatric disease rendering the subject unable to provide written informed consent or comply with evaluation procedures

• History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols

• Female of childbearing potential (apart of patient using adequate contraceptive measures), pregnant or breast feeding

• Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom follow-up evaluation is not assured)

• Limb surgery in the six months before randomization or planned before completion of the trial

• Known hypersensitivity to any of the individual components of PXT3003

• Porphyria